BACKGROUND AND OBJECTIVES Ocular myasthenia gravis (OMG) causes disabling ocular symptoms of ptosis and diplopia, but a validated disease-specific patient-reported outcome measure (PROM) has not been reported. We sought to validate a novel PROM for OMG, OMG Rating Scale Questionnaire (OMGRate-q), as a measure of visual functioning to support patient-centered care. METHODS This was a prospective study of patients aged 18 years and older with OMG receiving care at 3 medical centers (January 2022-October 2023). The 10-item OMGRate-q was administered, and response data were analyzed using exploratory factor analysis followed by Andrich rating scale model fitting. Poorly fitting items were eliminated, and the model was refit to produce the final items, item locations, and thresholds. Latent scores (theta) were estimated, test-retest reliability was established with repeat measures, and correlation with other myasthenia gravis PROMs was measured. RESULTS Of the 134 patients included in the study, 45 (33.6%) were women, 99 (73.9%) were White, and the median age (interquartile range [IQR]) was 64.6 years (52.6-73.9 years). A ptosis-related item showed significant item-trait deviation (p < 0.001) and was kept as a separate factor from the remaining diplopia-related items. After excluding this item, there were no misfitting items. Theta estimation for the diplopia scale ranged from -3.47 to 5.51 with median = -0.53 (IQR -2.33, 0.72). Test-retest reliability of the OMGRate-q diplopia subscale was high (intraclass correlation coefficient = 0.95 [95% CI 0.90-0.98]) and of the ptosis item was good (weighted κ = 0.56). No significant differences were observed in OMGRate-q diplopia subscale scores or the ptosis item between the 3 sites (diplopia p = 0.44; ptosis p = 0.32). OMGRate-q scores were moderately to highly correlated with the Myasthenia Gravis Quality of Life 15 questionnaire (n = 122; diplopia: r = 0.68, p < 0.001; ptosis: r = 0.48, p < 0.001) and Myasthenia Gravis Impairment Index (n = 130; diplopia: r = 0.76, p < 0.001; ptosis: r = 0.77, p < 0.001). OMGRate-q length was acceptable to most participants (125/130 [96.2%]), and the questionnaire was completed in 80.7 (±45.2) seconds. DISCUSSION The OMGRate-q is a valid and reliable disease-specific PROM for OMG that may be used to facilitate patient-centered research and care. However, the OMGRate-q emphasizes the impact of diplopia on visual functioning with a single separate item measuring ptosis. Future studies are needed to determine OMGRate-q responsiveness to disease-state changes and how to add measures of ptosis to this scale or whether a separate measure is needed.

中文翻译：

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眼重症肌无力评定量表问卷的多中心验证。


背景和目的 重症眼肌无力 （OMG） 导致上睑下垂和复视的致残性眼部症状，但尚未报道经过验证的疾病特异性患者报告结果测量 （PROM）。我们试图验证一种用于 OMG 的新型 PROM，即 OMG 评定量表问卷 （OMGRate-q），作为视觉功能的衡量标准，以支持以患者为中心的护理。方法 这是一项前瞻性研究，对象为在 3 个医疗中心接受护理的 18 岁及以上的 OMG 患者（2022 年 1 月至 2023 年 10 月）。对 10 项 OMGRate-q 进行管理，并使用探索性因子分析分析响应数据，然后进行 Andrich 评定量表模型拟合。剔除拟合不良的项，并对模型进行改装以生成最终项、项位置和阈值。估计潜在评分 （theta），通过重复测量建立重测信度，并测量与其他重症肌无力 PROM 的相关性。结果 在纳入研究的 134 例患者中，女性 45 例 （33.6%） ，白人 99 例 （73.9%），中位年龄 （四分位距 [IQR]）为 64.6 岁 （52.6-73.9 岁）。上睑下垂相关项目显示出显着的项目-性状偏差 （p < 0.001），并作为与其余复视相关项目不同的因素。排除此项后，没有不合适的项。复视量表的 θ 估计范围为 -3.47 至 5.51，中位数 = -0.53 （IQR -2.33， 0.72）。OMGRate-q 复视分量表的重测信度高 （组内相关系数 = 0.95 [95% CI 0.90-0.98]） 和上睑下垂项目的良好 （加权 κ = 0.56）。在 OMGRate-q 复视分量表评分或 3 个站点之间的上睑下垂项目方面未观察到显著差异 （复视 p = 0.44;上睑下垂 p = 0.32）。 OMGRate-q 评分与重症肌无力生活质量 15 问卷 （n = 122;复视： r = 0.68， p < 0.001;上睑下垂： r = 0.48，p < 0.001） 和重症肌无力损伤指数 （n = 130;复视： r = 0.76，p < 0.001;上睑下垂： r = 0.77，p < 0.001）呈中度至高度相关性。大多数参与者可以接受 OMGRate-q 长度 （125/130 [96.2%]），问卷在 80.7 （±45.2） 秒内完成。讨论 OMGRate-q 是一种有效且可靠的 OMG 疾病特异性 PROM，可用于促进以患者为中心的研究和护理。然而，OMGRate-q 强调复视对视觉功能的影响，通过一个单独的项目来测量上睑下垂。需要进一步的研究来确定 OMGRate-q 对疾病状态变化的反应性，以及如何将上睑下垂的测量添加到该量表中，或者是否需要单独的测量。